GX-H9, a long-acting recombinant human growth hormone codeveloped by  Handok and Genexine, has won the European Medicines Agency (EMA) designation as an orphan drug for growth hormone deficiency.

Handok and Genexine apply antibody-fusion technology (hyFc) to develop GX-H9, a next-generation growth hormone, for adult and pediatric patients. Unlike the conventional human growth hormones (HGH) that need to be administered daily, GX-H9, a long-acting recombinant HGH, only needs to be administered once a week or two weeks.

HGH is a protein hormone widely used to treat a child suffering from a decline in growth or developmental disability due to deficiency of growth hormones and hormone therapy for anti-aging and preventing lifestyle diseases.

Growth Hormone Deficiency (GHD) is a rare intractable disease caused by lack of hormone secretion due to pituitary failure, genetic deficiency, or tumor in the hypothalamus of the pituitary. Symptoms of adult patients include loss of muscle mass, increase in cholesterol, low bone density. It can also seriously affect children’s quality of life by causing dwarfism, a decline in growth, and delayed sexual development.

Orphan Drug Designation (ODD) of EMA is a variety of privileges given to developers that would enable prompt research and development of the treatment for intractable disease or other life-threatening diseases.

By winning the European ODD, GX-H9 will have the privilege of regulatory fee reduction, clinical trial protocol assistance, research funding aid, and 10 years of market exclusivity once it attains the permit.

“I am delighted with the EMA’s designation, following a similar move by the U.S. Food and Drug Administration (FDA) in 2016.” Handok CEO Kim Young-jin said. “With the affirmative data confirmed continuously through the clinical trial of the long-acting HGH, we will pursue the successful development of GX-H9 with Genexine.”

Handok and Genexine have developed GX-H9 since 2012. Korea Drug Development Fund selected the project as the recipient of support with its outstanding technology. The two co-developers are preparing for the multinational Phase 3 clinical trial of GX-H9 in the U.S. and Europe. Its Chinese partner, I-Mab, is also conducting phase 3 trials in its country.